Page 196 - Haematologica - Vol. 105 n. 6 - June 2020

L.K. Nielsen et al.
Table S2 and Online Supplementary Figure S2). Patients treated with MPR-R reported a statistically significant and clinically meaningful increase (small effect according to Cocks)24 in diarrhea (P<0.001, see Figure 3 and Online Supplementary Table S2). Peripheral neuropathy worsened in both arms (both P<0.001, see Online Supplementary Table S2), being clinically meaningful in patients treated with thalidomide only (Figure 3).
Clinically relevant differences between arms in HRQoL course during induction and maintenance
During treatment, clinically meaningful differences occurred between arms in 13 of 21 scales (Figure 3 and Online Supplementary Figure S2). Patients treated with MPT-T reported less diarrhea at all follow-up time points (difference between arms was of medium clinical effect, according to Cocks23), pain at T1, fatigue at T2, and insom- nia and appetite loss at T1 and T4. MPR-R treated patients reported better future perspective, physical and role func- tioning at T4, better cognitive functioning at T1 and T4, and body image at T3, compared to the patients treated with MPT-T. In addition, patients treated with MPT-T reported more side effects of treatment at T3 and T4 and
more constipation and peripheral neuropathy at all fol- low-up time points. According to the definition of Cocks, all the differences between the arms were of small clinical effect at the largest, except where stated differently (Online Supplementary Table S2).23
Changes in HRQoL within and between treatment arms during maintenance only
We performed an analysis of a subset of 242 patients who started and continued maintenance treatment for at least three months and of whom a T2 questionnaire was available: 95 of 146 patients who started with thalidomide maintenance therapy (65%) and 147 of 174 patients who started with lenalidomide maintenance therapy (84%). At the start of maintenance, there was already a significant difference in HRQoL in constipation, side effects of treat- ment and neuropathy (less in MPR treated patients) and diarrhea (less in MPT treated patients) (Online Supplementary Table S1). During maintenance treatment, a statistically significant reduction in appetite loss was reported in both arms (thalidomide P=0.003, lenalidomide P<0.001). In addition, during lenalidomide maintenance, a
Figure 2. Responders. The percentage of patients reaching a clinically relevant change in health-related quality of life (HRQoL), e.g. reaching the minimal important difference (MID) threshold for within group change during the induction phase (T2) and induction and maintenance phase together (T4). A significant difference between the arms with respect to the percentage of patients improving or deteriorating by more than the MID was observed for diarrhoea and peripheral neuropathy at T2 and for peripheral neuropathy at T4.
1654
haematologica | 2020; 105(6)